We won’t rest until we improve outcomes for patients with even the most resistant leukaemias

As haematological cancer outcomes and survival rates continue to improve worldwide thanks to earlier diagnosis and advances in treatment options, patients with various blood cancers can now live longer and better lives with their disease than ever before. (Blood Cancer UK; Chen 2022; Koehne 2022; Hochhaus 2022; Collins 2022)

The future for patients with acute lymphocytic leukaemia (ALL), acute myeloid leukaemia (AML), chronic lymphocytic leukaemia (CLL) and chronic myeloid leukaemia (CML) is certainly looking brighter than ever.

Yet, we still have work to do. With a growing and ageing population, the global incidence rate of haematological cancers continues to increase, imposing an ever greater treatment burden on patients and worldwide healthcare systems. (Sommer 2021; Roloff 2022)

Patients need more than just survival

Despite research breakthroughs and treatment innovations in recent years that are transforming previously devastating diseases into more chronic forms, patients continue to experience uncertain outcomes and relapse, with survival rates varying from one cancer to another. (Sommer 2022; Roloff 2022; García-Gutiérrez 2022; Collins 2022) While the five-year survival rates for CML and CLL are now over 85% and over 90% for children with ALL, the five-year survival rate for AML is still less than 50%, and overall survival of adults with ALL with standard chemotherapy is only 40-50%. (Blood Cancer UK; Advani 2022; Malczewska 2022)

Patients with disease relapse or progression face further treatment, with the risk of drug toxicity and resistance, as well as deteriorated physical and emotional well-being, and overall quality of life. (Sommer 2021; Kay 2022) This is particularly frequent in patients with AML, many of whom are refractory to their initial therapy or relapse following complete remission. (Roloff 2022) And while CML patients are now reaching similar survival to the general population, a major concern is the increasing development of comorbidities and poorer quality of life in these patients following diagnosis. (Sanz 2022; Iurlo 2022; García-Gutiérrez 2022; Hochhaus 2020)

This Blood Cancer Month reminds us that although improving survival remains a key objective, avoiding treatment side effects and relapse, achieving treatment-free remission where possible, and improving quality of life should be key considerations when managing haematological cancers. (Chen 2022; Iurlo 2022)

Patient stratification is essential to drive tailored treatment

The striking advances and improved outcomes in blood cancers have certainly changed our outlook on disease prognosis. However, with such a growing number of treatment options, therapeutic decision-making has become all the more complex. (Koehne 2022; Roloff 2022; García-Gutiérrez 2022)

It is therefore important, now more than ever, to integrate individual patient disease characteristics, as well as patient goals and preferences, into our treatment decision-making to optimise outcomes for all patients. (Roloff 2022; Stone 2022; García-Gutiérrez 2022; Hochhaus 2020) In heterogeneous cancers such as AML, the use of biomarker testing and scoring systems can help identify and stratify patients to better orient therapeutic decision-making. (Roloff 2022) Patient and disease stratification also gives us opportunities to develop more promising targeted therapies and immunotherapies to improve outcomes for those patients. (Roloff 2022; Ali 2022)

Understanding and monitoring disease biology will help optimise outcomes

For this reason, it is essential, whenever possible, to monitor patient response to treatment and assess their disease biology regularly and repetitively to better adapt and tailor (targeted) therapy to their individual needs, ensuring the best possible outcomes and preserving quality of life. (Stone 2022; Hochhaus 2022) Treatment-free remission (TFR) is now a real attainable goal for patients with CML in clinical centres with access to high-quality and ongoing monitoring. But such considerations remain limited in resource-poor settings, where monitoring is infrequent, availability of effective drugs is restricted, and patient survival alone remains the primary goal of treatment. (Hochhaus 2022)

More surprisingly however, observational studies have shown that European LeukemiaNet (ELN) monitoring recommendations are not consistently applied, even in high-income countries, leaving patients at higher risk of relapse. (García-Gutiérrez 2022) In patients with AML, assessment of measurable residual disease (MRD) remains challenging although several technologies are available. And despite clinician, patient, regulatory authority, industry, and researcher interest in MRD monitoring to help guide post-remission therapy and identify relapse, assays and reporting still lack standardisation. (Heuser 2021)

Rare but not forgotten

While leukaemias, like many blood cancers, tend to be rare forms of cancer, touching a limited number of people within Europe, the unmet need remains high. For certain patient groups, survival rates are extremely low. For example, for people over 65 years old diagnosed with AML, survival is five times lower than for younger patients (only around 6%). (Yi M 2020) There is much work to be done, and as an industry, we are committed to making investments for the populations that need it the most. 

In a world where blood cancer monitoring tools and treatments are now abundant, all patients should have access to high quality care. Through the "Time to Patient Access" project, the EFPIA Oncology Platform brings together stakeholders to identify stumbling blocks and elaborate practical solutions to improve patient access to the right treatment at the right time.

A joint effort

Blood Cancer Month (September 2022) is an opportunity to re-affirm our commitment to bringing new targeted therapies to all patients across Europe, and implement systematic patient monitoring and stratification to improve outcomes and quality of life for patients with leukaemia. This month reminds us of the challenges we still face to overcome stumbling blocks in effective patient care and ensure every patient has a chance of living a long and fulfilling life with one of these treatable cancers.

This month remind us that there are approx. 140 cancers originating in the blood-forming tissue. Despite all the scientific progress, these cancers still represent a significant health and economic burden for EU citizens. However, hematology is not mentioned in the Europe’s Beating Cancer Plan (EBCP). We therefore call for attention to hematology at least in the implementation of both the EBCP plan and the Cancer Mission to

  • enable the deployment of existing EBCP flagships with clear cut focus on hematology
  • accelerate research for early diagnosis and treatment, scaling up existing best practices
  • address the so-far disproportionately low investment in hematology via proper plan funding
  • implement  proper metrics for hematology in the Inequality Registry
  • enable EU-wide solutions for healthcare systems resilience and sustainability starting from hematology, given substantial resources and in-patient procedures required in hematology

With the EFPIA Oncology Platform, we engage in dialogue and forge partnerships with patient advocacy groups, healthcare professionals, academics, payers and policymakers to reduce the number of people suffering or still dying from blood cancer. Together, we can raise standards for patients across Europe.  Together we can avoid having hematology falling into the crack, ending up not being in cancer plans, nor rare disease plans.



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